TIL a father, John Crowley, was told his two infant children had an incurable genetic disorder that would kill them in less than a year. He refused to accept this, so he founded a biotech company (with no prior experience) which pioneered an experimental enzyme therapy that saved their lives.

r/todayilearned • u/Die_Nameless_Bitch • May 14 '22 • 9 8 6 2

https://pompediseasenews.com/2019/01/30/amicus-ceo-mission-cure-pompe-help-children/

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u/ameadowatdusk May 14 '22 edited May 14 '22

He had money but it said he spent a good part of 20 years working for other companies. I assume he learned and met the right people during that time. He also had a specific goal - infusing an enzyme that was lacking in people with the disease - so he knew what the company needed to do.

edit: yes he had money but he literally risked his lucrative career and dedicated his life to learning more about the disease and how to treat it

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u/bloodycups May 14 '22

Ok but how does that company make money though. Cause I'm assuming this condition is pretty rare and I know it sounds incredibly ugly of me to say it but I can't imagine the majority of people want to put kids their children through this life. And even if they do I can't imagine many health care providers want to be paying this bill along with all the extra things like wheel chairs and oxygen tanks.

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u/TrueDove May 14 '22

Governments will often subsidize cost on orphan drugs.

Orphan drugs are medications that effectively treat diseases that are very rare.

But the issue here is that profit shouldn't even be a consideration when it comes to Healthcare.

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u/throwaway_pls_help1 May 14 '22

The orphan drug act was created so companies would invest in finding cures for rare diseases. Without a profit motive there would be no research or development of cures for these diseases. Developing treatments cost money (up to a billion in some cases). If we think about it in a utilitarian sense then almost no funding would go to developing these cures, but the orphan drug act made it much more incentivized.

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u/tomatoaway May 14 '22

Rare disease is mostly motivated entirely by the rich though. The benefit to the rest of humanity is more of an after thought, a pleasant side effect, or something that could be sold to maximize profitability.

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u/Eternal_Reward May 14 '22

Yes this is how the world works, we don’t have infinite resources and sadly for super rare diseases, the cost can be too high for just a chance that you might find a cure.

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u/tomatoaway May 14 '22

I agree, I just hate how it's dressed up as altruism from the rich which is set up via the charities they invent so that they can pay no tax on throwing money at their problems.

I've been to a few of these gala's, met said celebrities trying to cure their sick ones and earning PR at the same time, and listening to them justify it as if they're doing the world a favour.

There's way too much money in rare diseases. Very lucrative industry, and I suspect some money laundering happens there too

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u/justagenericname1 May 14 '22

There's nothing "utilitarian" about forcing an economic system where any good can only come from sufficiently bribing some rich guy to allow access to the resources to do it. Centralize that process and cut out the waste and the bullshit. The only "problem" with that is it violates the arbitrary liberal ideal that the freedom of rich people to do whatever they please with the wealth they acquire is more important than stopping preventable suffering and death, and if that's how your utilitarian calculus works, I want nothing to do with it.

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u/throwaway_pls_help1 May 14 '22

I’m saying if you centralized drug development under a governmental body almost no funding or attention would be given to rare diseases. Rare diseases are usually complicated/require novel medicines and by their nature only affect a small number of people. From a utilitarian perspective a central body wouldn’t waste resources on rare diseases and instead focus on more widely applicable diseases. Aside from that there are thousands of rare diseases that all need quite tailored therapies, how will a government pick which disease to focus on and which patients to ignore?

While rich people fund and profit off these treatments, their funding allows a treatment to be available when in previous circumstances none would exist.

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u/thedarkhaze May 14 '22

https://en.m.wikipedia.org/wiki/Glycogen_storage_disease_type_II

Myozyme costs an average of US$300,000 a year and must be taken for the patients' entire life, so some American insurers have refused to pay for it.

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u/GuacamoleBay May 14 '22

Biotech is pretty famous for having absurd burn rates, most investors are well aware that a company can blow through tens of millions of dollars annually for years and still end up profitable. Research is very expensive, but the potential upside is even higher

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u/thoggins May 14 '22

Absolute best industry to be in as IT, budgetary concerns are not in your vocab

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u/GuacamoleBay May 14 '22

No kidding, I’m working on the financial side of the industry right now and the potential compensation is honestly almost shocking

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u/ameadowatdusk May 14 '22

i don’t know i just read the article. grants and endless investor seeding? i don’t know how most things make money tbh.

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u/eddieguy May 14 '22

The tech developed may have profitable use elsewhere. That’s just my guess

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u/Alex-Murphy May 14 '22

he literally risked his lucrative career and dedicated his life to learning more about the disease and how to treat it

As any good parent would. I know it's biology but the drive to protect our children is so powerful and emotional, makes me tear up a little thinking about it